EP3924481A4 - Zusammensetzungen und verfahren zur behandlung von hämoglobinopathien - Google Patents

Zusammensetzungen und verfahren zur behandlung von hämoglobinopathien Download PDF

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Publication number
EP3924481A4
EP3924481A4 EP20755892.5A EP20755892A EP3924481A4 EP 3924481 A4 EP3924481 A4 EP 3924481A4 EP 20755892 A EP20755892 A EP 20755892A EP 3924481 A4 EP3924481 A4 EP 3924481A4
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EP
European Patent Office
Prior art keywords
hemoglobinopathies
compositions
treatment
methods
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP20755892.5A
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English (en)
French (fr)
Other versions
EP3924481A1 (de
Inventor
Ian SLAYMAKER
Nicole GAUDELLI
Yi Yu
Bernd ZETSCHE
David A. BORN
Seung-Joo Lee
Michael Packer
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Beam Therapeutics Inc
Original Assignee
Beam Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Beam Therapeutics Inc filed Critical Beam Therapeutics Inc
Publication of EP3924481A1 publication Critical patent/EP3924481A1/de
Publication of EP3924481A4 publication Critical patent/EP3924481A4/de
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/15—Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/18—Erythrocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4717—Plasma globulins, lactoglobulin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0641—Erythrocytes
    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Animal Behavior & Ethology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
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  • Developmental Biology & Embryology (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Diabetes (AREA)
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  • Toxicology (AREA)
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  • Enzymes And Modification Thereof (AREA)
EP20755892.5A 2019-02-13 2020-02-13 Zusammensetzungen und verfahren zur behandlung von hämoglobinopathien Pending EP3924481A4 (de)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US201962805277P 2019-02-13 2019-02-13
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US201962931747P 2019-11-06 2019-11-06
US201962931722P 2019-11-06 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US202062966526P 2020-01-27 2020-01-27
PCT/US2020/018193 WO2020168133A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating hemoglobinopathies

Publications (2)

Publication Number Publication Date
EP3924481A1 EP3924481A1 (de) 2021-12-22
EP3924481A4 true EP3924481A4 (de) 2023-01-25

Family

ID=72043877

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20755892.5A Pending EP3924481A4 (de) 2019-02-13 2020-02-13 Zusammensetzungen und verfahren zur behandlung von hämoglobinopathien

Country Status (8)

Country Link
US (7) US20220235347A1 (de)
EP (1) EP3924481A4 (de)
CN (3) CN116497067B (de)
AU (4) AU2020223060B2 (de)
CA (3) CA3236512C (de)
SG (1) SG11202107045PA (de)
WO (1) WO2020168133A1 (de)
ZA (4) ZA202104676B (de)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3443081A4 (de) 2016-04-13 2019-10-30 Duke University Crispr/cas9-basierte repressoren zur in-vivo-ausschaltung von gen-targets und verfahren zur verwendung
EP4275747A3 (de) 2016-07-19 2024-01-24 Duke University Therapeutische anwendungen von cpf1-basierter genomeditierung
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3850088A4 (de) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Zusammensetzungen und verfahren zur verbesserten basisbearbeitung
AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
CN114040977B (zh) 2019-01-31 2024-09-03 比姆医疗股份有限公司 非靶脱氨反应减低的核碱基编辑器和用于定性核碱基编辑器的测定
SG11202108346WA (en) 2019-02-13 2021-08-30 Beam Therapeutics Inc Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
AU2020223314A1 (en) 2019-02-13 2021-08-12 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
AU2020221366A1 (en) * 2019-02-13 2021-08-26 Beam Therapeutics Inc. Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence
CA3236512C (en) 2019-02-13 2026-02-24 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
EP3942043A2 (de) 2019-03-19 2022-01-26 The Broad Institute, Inc. Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN115279398B (zh) 2019-09-27 2025-07-04 比姆医疗股份有限公司 治疗液态癌症的组合物和方法
BR112022015969A2 (pt) * 2020-02-13 2022-10-25 Beam Therapeutics Inc Composições e métodos para enxerto de células editadas de base
WO2021189110A1 (en) * 2020-03-25 2021-09-30 University Of Tasmania Dna altering proteins and uses therefor
WO2021243289A1 (en) * 2020-05-29 2021-12-02 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)
US12576151B2 (en) 2020-09-25 2026-03-17 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20230383270A1 (en) * 2020-10-12 2023-11-30 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
WO2022232839A1 (en) * 2021-04-30 2022-11-03 The Board Of Trustees Of The Leland Stanford Junior University Methods for improved production of primary cd34+ cells
US20230287456A1 (en) * 2021-09-10 2023-09-14 Arbor Biotechnologies, Inc. Compositions comprising a cas12i polypeptide and uses thereof
EP4405478A1 (de) 2021-09-20 2024-07-31 Alnylam Pharmaceuticals, Inc. Inhibinuntereinheits-beta-e (inhbe)-modulatorzusammensetzungen und verfahren zur verwendung davon
CN117925585A (zh) * 2022-04-07 2024-04-26 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
WO2023196588A2 (en) * 2022-04-08 2023-10-12 Beam Therapeutics Inc. Hemoglobin g-makassar binding polypeptides and antibodies and methods of using the same
WO2023220570A2 (en) * 2022-05-09 2023-11-16 Mammoth Biosciences, Inc. Engineered cas-phi proteins and uses thereof
EP4543469A2 (de) * 2022-06-27 2025-04-30 Beam Therapeutics Inc. Zusammensetzungen und verfahren zur nicht-genotoxischen zellkonditionierung
AU2023375263A1 (en) * 2022-11-02 2025-04-10 Incisive Genetics, Inc. Compositions and methods for preventing, ameliorating, or treating sickle cell disease and compositions and methods for disrupting genes and gene segments
CN118871578A (zh) * 2023-05-24 2024-10-29 锐正基因(苏州)有限公司 用于碱基编辑的脱氨酶及其变体
WO2025024285A1 (en) * 2023-07-21 2025-01-30 Mammoth Biosciences, Inc. Compositions for the modification of the human c9orf72 gene
WO2025117698A1 (en) * 2023-12-01 2025-06-05 Massachusetts Institute Of Technology Ribozyme-enhanced rna trans-splicing
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025231247A1 (en) * 2024-05-02 2025-11-06 University Of Massachusetts Editing of aatd-related genes using a nme2 base editor

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006498A2 (en) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2018027078A1 (en) * 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US20180223313A1 (en) * 2015-10-02 2018-08-09 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Lentiviral protein delivery system for rna-guided genome editing
US20180237768A1 (en) * 2015-06-18 2018-08-23 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2019018383A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
WO2020168051A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2591356A1 (de) 2010-07-09 2013-05-15 Ecole Polytechnique Fédérale de Lausanne (EPFL) Verfahren zur in-vitro-überwachung von neuronalen erkrankungen sowie anwendung
DK2836226T3 (en) * 2012-02-24 2017-09-18 Hutchinson Fred Cancer Res COMPOSITIONS AND PROCEDURES FOR TREATING HEMOGLOBINOPATHY
JP2015529466A (ja) * 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
EP2931898B1 (de) 2012-12-12 2016-03-09 The Broad Institute, Inc. Technische konzeption und optimierung von systemen, verfahren und zusammensetzungen zur sequenzmanipulation funktionellen domänen
ES3029138T3 (en) 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2015148863A2 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
BR112017007765B1 (pt) 2014-10-14 2023-10-03 Halozyme, Inc Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
JP7044373B2 (ja) 2015-07-15 2022-03-30 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
IL313038A (en) 2016-03-14 2024-07-01 Editas Medicine Inc Methods and preparations related to CRISPR/CAS - for the treatment of diseases in the hemoglobin cell
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
IL268058B2 (en) 2017-01-20 2023-09-01 Magenta Therapeutics Inc Compositions and methods for depleting cd137 plus cells
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CN108823202A (zh) * 2017-06-15 2018-11-16 中山大学 用于特异性修复人hbb基因突变的碱基编辑系统、方法、试剂盒及其应用
CA3064601A1 (en) 2017-06-26 2019-01-03 President And Fellows Of Harvard College Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
US10882915B2 (en) 2017-10-24 2021-01-05 Magenta Therapeutics, Inc. Compositions and methods for the depletion of CD117+ cells
CA3085338A1 (en) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
CN110157727A (zh) * 2017-12-21 2019-08-23 中国科学院遗传与发育生物学研究所 植物碱基编辑方法
AR114014A1 (es) * 2017-12-22 2020-07-08 Inst Genetics & Developmental Biology Cas Sistema de edición de bases y método basado en proteína cpf1
US11268092B2 (en) * 2018-01-12 2022-03-08 GenEdit, Inc. Structure-engineered guide RNA
EP3762496A2 (de) 2018-03-07 2021-01-13 Editas Medicine, Inc. Systeme und verfahren zur behandlung von hämoglobinopathien
EP3781585A4 (de) 2018-04-17 2022-01-26 The General Hospital Corporation Empfindliche in-vitro-tests für substratpräferenzen und stellen für nukleinsäurebindungs-, -modifizierungs- und spaltungsmittel
EP3790964A4 (de) 2018-05-11 2022-06-08 Beam Therapeutics, Inc. Verfahren zur unterdrückung von pathogenen mutationen unter verwendung von programmierbaren basiseditorsystemen
KR20250133482A (ko) 2018-05-11 2025-09-05 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN112805379B (zh) * 2018-08-03 2024-08-20 比姆医疗股份有限公司 多效应核碱基编辑器和使用其修饰核酸靶序列的方法
CN110835629B (zh) * 2018-08-15 2022-07-26 华东师范大学 一种新型碱基转换编辑系统的构建方法及其应用
US20230021641A1 (en) 2018-08-23 2023-01-26 The Broad Institute, Inc. Cas9 variants having non-canonical pam specificities and uses thereof
GB201815670D0 (en) 2018-09-26 2018-11-07 Univ Oxford Innovation Ltd Protein editing
EP3887521A1 (de) 2018-11-29 2021-10-06 Editas Medicine, Inc. Systeme und verfahren zur behandlung von hämoglobinopathien
EP3921417A4 (de) 2019-02-04 2022-11-09 The General Hospital Corporation Varianten von adenin-dna-basen mit reduzierter off-target-rna-editierung
CA3128881A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
CA3236512C (en) 2019-02-13 2026-02-24 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
AU2020221366A1 (en) 2019-02-13 2021-08-26 Beam Therapeutics Inc. Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence
CN110042124A (zh) 2019-04-25 2019-07-23 国家卫生健康委科学技术研究所 基因组碱基编辑增加人红细胞中胎儿血红蛋白水平的试剂盒及应用
CN112175927B (zh) * 2019-07-02 2023-04-18 上海科技大学 一种碱基编辑工具及其用途
WO2021041945A2 (en) 2019-08-29 2021-03-04 Beam Therapeutics Inc. Compositions and methods for non-toxic conditioning
EP4028026A4 (de) 2019-09-09 2023-09-06 Beam Therapeutics, Inc. Neuartige nukleobase-editoren und verfahren zur verwendung derselben
BR112022015969A2 (pt) 2020-02-13 2022-10-25 Beam Therapeutics Inc Composições e métodos para enxerto de células editadas de base
EP4229195A4 (de) 2020-10-14 2025-08-13 Beam Therapeutics Inc Zusammensetzungen und verfahren zur behandlung der glycogenspeicherkrankheit vom typ 1a

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006498A2 (en) * 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
US20180237768A1 (en) * 2015-06-18 2018-08-23 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US20180223313A1 (en) * 2015-10-02 2018-08-09 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Lentiviral protein delivery system for rna-guided genome editing
WO2018027078A1 (en) * 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
WO2019018383A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
WO2020168051A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MARIA ROSA LIDONNICI ET AL: "Gene therapy and gene editing strategies for hemoglobinopathies", BLOOD CELLS, MOLECULES & DISEASES, vol. 70, 1 May 2018 (2018-05-01), US, pages 87 - 101, XP055597660, ISSN: 1079-9796, DOI: 10.1016/j.bcmd.2017.12.001 *
YAN DAQI ET AL: "High-efficiency and multiplex adenine base editing in plants using new TadA variants", vol. 14, no. 5, 1 May 2021 (2021-05-01), pages 722 - 731, XP093006106, ISSN: 1674-2052, Retrieved from the Internet <URL:http://dx.doi.org/10.1016/j.molp.2021.02.007> DOI: 10.1016/j.molp.2021.02.007 *

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